Clinical Trials

It can take as long as 12 years for a new therapy to be made available to patients.

One of the most critical stages of the process is clinical trials.

Helping Patients

Clinical development is the most time intensive and expensive part of the medical research and development process. It accounts for 45 to 75 percent of the $50 million average cost of bringing a new therapy to market.

Trials conducted during clinical development generate thousands of pieces of data and are generally used by regulatory agencies, such as the U.S. Food and Drug Administration (FDA), to determine approval for use of the therapy and how it will be used.

These trials involve hundreds to several thousands of volunteer participants.

For patients suffering from a condition that is currently untreatable, participation in a clinical trial may give them access to potential new therapies. In addition, volunteers are helping others by contributing to medical research.

Phases of Development

Clinical Development Process

An experimental therapy is first tested in the laboratory and in animal studies. After this “pre-clinicaI” testing, and only if shown to meet certain safety criteria and to have value as a potential new therapy, the therapy advances to clinical testing in humans. The Phases of Clinical Development:

Phase 1 clinical trials mark the first time an experimental therapy is administered to humans. Phase 1 trials usually focus on ensuring the therapy is safe to use in people, rather than how effective it may be as a treatment for a given disease. During this phase, escalating doses of the experimental therapy are given to a small number of study participants so that researchers can measure the body’s response, including how it is absorbed, its duration in the blood stream, and which dosage levels are safe and well-tolerated.

Phase 2 trials generally assess the effectiveness of an experimental therapy at treating a specific illness or medical condition. Information about the experimental therapy’s safety, side effects and potential risks are also collected. In this phase, researchers work to determine the most effective dosages for the experimental therapy and the most appropriate method of delivery, such as tablets, extended release capsules, infusions or injections. Phase 2 clinical trials involve a larger number of participants, typically up to several hundred, who usually have the medical condition that the therapy is intended to treat. These trials test the results of earlier trials in much larger groups and gather additional information on the effectiveness and safety of the therapy. This phase will usually involve several hundred to several thousand participants across multiple study locations. These trials are often randomized, where participants are arbitrarily allocated to receive the experimental therapy, placebo or another therapy (a “comparator”), and “double-blinded,” in which neither the investigator nor the participant are aware if the therapy given is the true experimental therapy, placebo (medication with no active ingredients) or another therapy (a “comparator”). Phase 3 trials generally provide the primary basis for the benefit-risk assessment for the new therapy and much of the core information about the therapy that is analyzed for inclusion in final labeling, if approved by the regulatory authority.

Phase 4 trials — also called “post-marketing studies” — are conducted after regulatory approval and are critical to informing the ongoing use of the therapy. Through such trials, researchers collect additional information about longer-term risks, benefits and optimal use. These trials often involve thousands of participants and may continue for many years.

Notice: The information provided on this page is for general information only. It is not intended to provide medical advice or guidance, and should not be used for that purpose. Medical use of any product should be strictly in accordance with the approved prescribing information.

Registration

Filing an application for registration with the country’s health regulatory authority is the next step in bringing a potential new therapy to patients. In the U.S., a New Drug Application (NDA) is filed with the U.S. Food and Drug Administration (FDA). In Europe, a Market Authorization Application (MAA) is filed with the European Agency for the Evaluation of Medicinal Products (EMEA). A description of the therapy’s manufacturing process, along with quality data and trial results from all pre-clinical and clinical trials, are provided in order to demonstrate the safety and effectiveness of the new therapy. If approval is granted, the new therapy can then be sold for use as detailed in the final approval label granted by the regulatory authority.

Who Regulates Stem Cell Treatment Protocols and Therapy?

There are two aspects to consider regarding the use of stem cell therapies in the U.S. and potential host countries outside of the U.S.

The first is the existing legal structure surrounding research, and the second is the regulatory framework regarding application of that research to treat patients.

The science teams we associate with adhere to the guidelines promulgated by the U.S. Food and Drug Administration (FDA) under the U.S. Department of Health and Human Services, Code of Federal Regulation, Part 1271 entitled “Regulation of Human Cells, Tissues, and Cellular and Tissue Based Products”.

The FDA makes a clear distinction of minimally manipulated cells and autologous transplantation from other cell types when it states in Part 1271, that “you are not required to comply with the requirements of this part if you are an establishment that removes human cells, tissues and cellular and tissue-based products from an individual and implants such products into the same individual during the same surgical procedure”. What this implies is that minimally manipulated and autologous cell therapy should not have regulations that are in place for other human cells, tissues, tissue-based products and drugs.

The science teams we associate with follow Part 1271 for adult autologous stem cell transplant.

Adult HSC (Hematopoietic Stem Cell) transplants under practice and approved by FDA over the last 30 years include the following examples of diseases:

Malignant:

Acute myeloid leukemia (AML)

Chronic myeloid leukemia (CML)

Acute lymphoblastic leukemia (ALL)

Hodgkin lymphoma (relapsed, refractory)

Non-Hodgkin (relapsed or refractory) lymphoma

Neuroblastoma

Ewing sarcoma

Multiple Myeloma

Myelodysplastic syndromes

Gliomas, other solid tumors

Non-malignant

Thalassemia

Sickle Cell Anemia

Aplastic anemia

Fanconi anemia

Immune deficiency syndromes

Inborn errors of metabolism

Regarding patient application in their host countries, prohibitions are generally in place regulating the use of embryonic stem cell. For the most part, the science teams we associate with do not conduct research or treat patients with embryonic stem cells.

Clinical Trial Participation

The science teams we associate with create policies and case studies to ensure and demonstrate their ethical development of new medicines. All clinical research must be reviewed by a qualified Independent Ethics Committee or Institutional Review Boards.

The science teams we associate with report their patient findings based on:

Disease and length of time after transplant: 1 year, 3 year.

Type of donor who provided the cells for transplant:

Autologous (the patient’s cells)

Related allogeneic (a patient’s sibling or another family member’s cells)

Unrelated allogeneic (a volunteer donor’s cells)

The cells used for transplant can be from bone marrow, peripheral blood, umbilical cord blood or adipose tissues. Outcome data shows survival information for transplant patients with a similar disease. The information cannot predict the outcome of any one person. One person’s response to transplant may be very different than another person’s response. The number of patients evaluated can greatly affect the long-term survival estimate.

When only a small number of patients are observed, the estimated outcomes can vary widely.

When a large number of patients are observed, the estimate is more reliable. Long-term survival outcomes only provide a partial picture. A patient’s specific medical condition, health, and previous treatments and other factors must also be considered. Patients should refer questions to their doctor, transplant center staff or another health care professional.

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